# A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2

> **NCT04221451** · PHASE3 · TERMINATED · sponsor: **Genzyme, a Sanofi Company** · enrollment: 75 (actual)

## Conditions studied

- Tay-Sachs Disease
- Sandhoff Disease

## Interventions

- **DRUG:** venglustat GZ402671
- **DRUG:** placebo

## Key facts

- **NCT ID:** NCT04221451
- **Lead sponsor:** Genzyme, a Sanofi Company
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE3
- **Study type:** INTERVENTIONAL
- **Status:** TERMINATED
- **Start date:** 2020-06-29
- **Primary completion:** 2024-12-26
- **Final completion:** 2024-12-26
- **Target enrollment:** 75 (ACTUAL)
- **Why stopped:** Discontinued based on the absence of positive trends on clinical endpoints.
- **Last updated:** 2026-01-28


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT04221451

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT04221451, "A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2". Retrieved via AI Analytics 2026-06-03 from https://api.ai-analytics.org/clinical/NCT04221451. Licensed CC0.

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*[Clinical trials dataset](/datasets/clinical-trials) · CC0 1.0*